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Results for “Genetic Diseases, Inborn”

16,203 trials

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Showing 20 of 16,203 results

Not applicableLooking for participantsNCT06610019
What this trial is testing

Cardiovascular Multimodality Imaging Study

Who this might be right for
Non-ischemic CardiomyopathyCardiomyopathiesHypertrophic Cardiomyopathy+7 more
Montefiore Medical Center 5,000
Not applicableStudy completedNCT04892628
What this trial is testing

Effects of Progressive Resistance Training Compared With Aerobic Training in Patients With Hemophilia

Who this might be right for
Quality of LifeMuscle Weakness
Dow University of Health Sciences 60
Post-approval studies (Phase 4)Study completedNCT06925100
What this trial is testing

Investigate the Effectiveness and Safety of Bempedoic Acid in Patients With Primary Hypercholesterolemia or Mixed Dyslipidemia in Taiwan

Who this might be right for
Primary Hypercholesterolemia or Mixed Hyperlipidemia
Daiichi Sankyo 180
Testing effectiveness (Phase 2)Study completedNCT01055847
What this trial is testing

Aztreonam for Inhalation (AI) in Patients With Cystic Fibrosis & P. Aeruginosa Infection

Who this might be right for
Cystic FibrosisCFLung Infection+1 more
Gilead Sciences 105
Post-approval studies (Phase 4)Study completedNCT01064947
What this trial is testing

Safety and Efficacy Study of Altabax Ointment in the Treatment of Secondarily Infected Atopic Dermatitis

Who this might be right for
Atopic DermatitisSecondary Infection
Derm Research, PLLC 29
Not applicableNot Yet RecruitingNCT07333300
What this trial is testing

Exploratory Clinical Investigation on Neurosensory Responses Via EEG Headband in Adults With Mild to Moderate Atopic Dermatitis After Application of Marketed Product Compared With Placebo (Water Application).

Who this might be right for
Atopic Dermatitis (AD)
NovoBliss Research Pvt Ltd 20
Testing effectiveness (Phase 2)UnknownNCT02386267
What this trial is testing

L-leucine in Diamond Blackfan Anemia Patients

Who this might be right for
Diamond Blackfan Anemia
Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache 30
Testing effectiveness (Phase 2)Study completedNCT01412151
What this trial is testing

Creatine Safety & Tolerability in Huntington's Disease

Who this might be right for
Huntington's Disease (HD)
Massachusetts General Hospital 10
Large-scale testing (Phase 3)Study completedNCT04058353
What this trial is testing

VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Who this might be right for
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated 271
Not applicableLooking for participantsNCT06935253
What this trial is testing

Large Language Models To Improve the Quality of Care of Cardiology Patients

Who this might be right for
Hypertrophic Cardiomyopathy (HCM)CardiomyopathyGenetic Disease+1 more
Stanford University 12
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07341022
What this trial is testing

STEM CELL MOBILIZATION WITH HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE

Who this might be right for
Sickle Cell Disease
Hospital Israelita Albert Einstein 12
Large-scale testing (Phase 3)Study completedNCT00426153
What this trial is testing

Octreotide in Severe Polycystic Liver Disease

Who this might be right for
Polycystic Kidney, Autosomal DominantPolycystic Liver DiseaseHepatomegaly+3 more
Mayo Clinic 42
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Post-approval studies (Phase 4)Study completedNCT00134745
What this trial is testing

Defining the Optimal Hormonal Replacement Therapy in Turner Syndrome

Who this might be right for
Turner Syndrome
Line Cleemann 43
Testing effectiveness (Phase 2)Active Not RecruitingNCT04980872
What this trial is testing

The Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

Who this might be right for
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Merck Sharp & Dohme LLC 60
Testing effectiveness (Phase 2)Study completedNCT02579239
What this trial is testing

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)

Who this might be right for
Limb-Girdle Muscular DystrophiesFacioscapulohumeral Muscular Dystrophy
aTyr Pharma, Inc. 18
Not applicableUnknownNCT01791075
What this trial is testing

Comparing Endoglide to Endoserter for DSAEK Graft Insertion

Who this might be right for
Fuch's Endothelial DystrophyBullous Keratopathy, Unspecified Eye
University Health Network, Toronto 42
Not applicableStudy completedNCT05388357
What this trial is testing

AI Quantitative Coronary Angiography Versus Optical Coherent Tomography Guidance for Coronary Stent Implantation

Who this might be right for
Coronary VesselsProcedure PCI
Asan Medical Center 400
Post-approval studies (Phase 4)Study completedNCT00144781
What this trial is testing

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 34
Not applicableEnded earlyNCT03823443
What this trial is testing

COPD Exacerbation Blood and Urine Biomarkers Study

Who this might be right for
Chronic Obstructive Pulmonary DiseaseAcute Exacerbation CopdAlpha-1 Anti-trypsin Deficiency
Columbia University 7
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